On June 3, 2021 Kyowa Kirin Co., Ltd. (President and CEO: Masashi Miyamoto, "Kyowa Kirin", TSE: 4151) reported that it will make a change in its organization as of July 1, 2021 (Press release, Kyowa Hakko Kirin, JUN 3, 2021, View Source [SID1234583433]).

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Outline of the change:
R&D Quality Assurance Department will be newly established within the Quality Division.

Purpose of the organizational change:
By integrating quality assurance activities of clinical and non-clinical studies into the Quality Division, execute drug development and post-marketing clinical trials activities in a more reliable organizational structure.

On June 2, 2021 Pacylex Pharmaceuticals, an oncology company unlocking a new approach to cancer therapy, and Greenfire Bio, a new Life Science development and investment company, reported the closing of Series A financing for Pacylex (Press release, Pacylex Pharmaceuticals, JUN 2, 2021, View Source [SID1234645063]). These funds will be used to support the initial Phase 1 clinical investigation of PCLX-001, a first-in-class N-myristoyltransferase (NMT) inhibitor, in Diffuse Large B-Cell Lymphoma and solid tumor patients. Pacylex is leading the development of novel therapies targeting the biological process of myristoylation in cancer.

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"We are excited to be a catalyst for this new innovation in oncology" said Ajit Gill, CEO of Greenfire Bio. "Our goal is to build a portfolio of potential breakthroughs in medicine, and we look forward to seeing PCLX-001 move into the clinic."

"The support from Greenfire Bio is essential for our transformation into a clinical stage company", said Michael Weickert, CEO of Pacylex. "Pioneering a new target and first-in-class therapy is extraordinarily important to expand cancer treatment options and improve patient outcomes. We are delighted to find the right investor with an appreciation for this groundbreaking work."

Clinical site preparations are underway for the open label, dose escalation, Phase 1 clinical trial, principally to evaluate the safety of PCLX-001. The study will enroll 20-30 patients and the Company anticipates that enrollment will begin within the next month. A No Objection Letter from Health Canada was received by Pacylex on March 8, 2021, authorizing the planned Phase 1 Trial of PCLX-001 in relapsed/refractory B-cell Non-Hodgkin Lymphoma and advanced solid malignancies. PCLX-001 is believed to be the first NMT inhibitor that will be clinically tested. Three principal investigators will oversee the clinical study at three clinical sites in Canada: Dr. John Kuruvilla at Princess Margaret Cancer Centre in Toronto, Dr. Randeep Sangha at the Cross Cancer Institute in Edmonton and Dr. Laurie Sehn at the British Columbia Cancer Center in Vancouver.

PCLX-001

PCLX-001 is a small molecule, first-in-class NMT inhibitor, originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness funded by Wellcome Trust. Pacylex is developing PCLX-001, which has excellent oral bioavailability, to treat leukemia and lymphoma. PCLX-001 selectively kills cancer cells and completely regresses (eliminates) tumors in animal models of acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL) and Burkitt lymphoma (BL). PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the level of NMT2 is correlated with survival, suggesting an important biological role in these cancers. In tests using cultured cancer cells in vitro, PCLX-001 is at least ten times as potent as ibrutinib (Imbruvica) and dasatinib (Sprycel), two clinically approved drugs currently used to treat hematologic malignancies.

On June 2, 2021 Aptamer Science reported the company signed a joint research agreement with bio venture company PINOTBIO to develop anticancer drugs based on Aptamer Drug Conjugate (ApDC) (Press release, Aptamer Sciences, JUN 2, 2021, View Source;idx=127 [SID1234644070]).

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Both companies plan to accelerate the development of innovative targeted anti-cancer drugs that go beyond the limitations of existing ‘antibody-drug conjugate (ADC)’ technology by linking their respective core technologies, aptamer discovery technology and linker-drug conjugation technology. am.

The ‘Antibody-Drug Conjugate (ADC)’ technology, which has recently been in the spotlight, has difficulty controlling the conjugation of antibodies and drugs, and has limitations in penetrating cancer tissue due to its molecular size, so there is a steady demand for the development of technology to complement this. The situation continues.

Aptamers, a nucleic acid-based material that is attracting attention as a next-generation drug material, show high selectivity and binding ability to targets and have a molecular size that facilitates tissue penetration. In addition, the ability to design chemical reactions that can control the number and location of drug conjugates is considered an advantage of ApDC technology.

Under this agreement, the two companies plan to conduct joint research aimed at developing targeted anticancer drugs for glioblastoma and pancreatic cancer, which have high unmet medical needs. To this end, Aptamer Science will provide optimized cancer cell-targeting aptamers, and Pinobio will provide He will be responsible for conjugation and efficacy evaluation of tamers and drugs.

Dong-il Han, CEO of Aptamer Science, said, "Through joint research with Pinobio, which has its own linker-drug technology, we want to develop a new type of targeted anti-cancer drug that overcomes the limitations of existing ADC technology." He added, "The collaboration between the two companies is a meaningful clinical trial." "We will do our best to ensure that the outcome is favorable," he said.

Doo-young Jeong, CEO of Pinobio, said, "Through joint research with Aptamer Science, we are able to develop ApDC technology using aptamers, which have characteristics differentiated from antibodies, which are existing drug carriers, so we expect to quickly derive innovative candidates and ADC drug technology. "We expect to be able to present a new paradigm," he said.

On June 2, 2021 Sysmex Inostics, Inc., a global leader in the liquid biopsy revolution for oncology, reported that it is presenting the poster entitled, "Ultra-sensitive detection and quantification of human papillomavirus (HPV) DNA in the plasma of patients with oropharyngeal squamous cell carcinoma (OPSCC) enrolled in the OPTIMA 2 treatment de-escalation trial" at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting, June 4-8, 2021 (Press release, Sysmex Inostics, JUN 2, 2021, View Source [SID1234586884]). The featured data show that SafeSEQ cfHPV-DNA Test (HPV-SEQ) exhibits robust quantitative detection of cell-free HPV DNA (cfHPV-DNA) across a broad dynamic range, enabling high-resolution monitoring for patients with HPV+ OPSCC, a type of head and neck cancer. The ASCO (Free ASCO Whitepaper) presentation coincides with the launch of HPV-SEQ as the newest CLIA validated assay in the Sysmex Inostics portfolio of ultra-sensitive SafeSEQ NGS panels.

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HPV 16 and HPV 18 are the two most common high-risk HPV strains, and persistent infections can lead to HPV-related cancers, including cervical, anal, and head and neck cancers. HPV driven tumors are increasing in prevalence, especially in patients presenting with head and neck cancer.1 cfHPV-DNA can be measured in patients’ plasma as a non-invasive surrogate of tumor burden and can facilitate precise tracking of disease response throughout treatment.

Sysmex Inostics has introduced HPV-SEQ, an ultra-sensitive blood-based liquid biopsy solution for identifying and accurately quantifying circulating HPV 16 and HPV 18 DNA in patients with HPV-related cancers.

As patients with HPV-driven tumors often have a good prognosis, clinical investigators have recently explored new strategies for treatment de-escalation to avoid unnecessary side-effects caused by overtreatment. Important clinical data for HPV-SEQ was generated during the recent OPTIMA 2 phase II trial (NCT03107182) investigating induction chemoimmunotherapy followed by risk/response stratified de-escalated locoregional therapy for patients with HPV+ OPSCC. During the trial, HPV-SEQ was employed to evaluate levels of cfHPV-DNA alongside patients’ radiographic response to therapy in order to assess the future utility in guiding treatment de-escalation strategies. HPV-SEQ showed robust quantitative detection of HPV 16/18 across a broad dynamic range over five orders of magnitude with low quantitative variability. Importantly, a high correlation was observed between dynamic changes in patients’ cfHPV DNA levels and radiographic responses following induction therapy.

"HPV-SEQ exhibits robust quantitative detection of HPV, even when only a few copies are present, thus potentially enabling precise molecular monitoring of patients’ therapy response," said Dr. Nishant Agrawal, Professor of Surgery at the University of Chicago. Dr. Agrawal added, "We see the HPV-SEQ test becoming an important tool for refining patient treatment strategies and accelerating the development of novel de-escalation approaches for the treatment of HPV-associated OPSCC."

Poster number 6048, "Ultra-sensitive detection and quantification of HPV DNA in the plasma of patients with oropharyngeal squamous cell carcinoma (OPSCC) enrolled in the OPTIMA 2 treatment de-escalation trial," presented by Hillary Sloane, PhD, Associate Director of Medical & Scientific Affairs at Sysmex Inostics, will be available June 4 through June 8, 2021 during the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting.

Currently, HPV-SEQ is being used in prospective studies to further evaluate the kinetics of cfHPV-DNA as a predictor of response to therapy in patients with HPV+ OPSCC and other HPV-related cancers. HPV-SEQ is CLIA-validated and available to support clinical trials through the Sysmex Inostics turnkey testing service in their CLIA lab located in Baltimore, Maryland.

On June 2, 2021 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported an update on recent progress with its two pipeline assets, paxalisib and EVT801 (Press release, Kazia Therapeutics, JUN 2, 2021, View Source [SID1234584522]).

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Key Points

GBM AGILE pivotal study of paxalisib is recruiting ahead of expectations, with almost 25 sites now open to the paxalisib arm.
Paxalisib phase II study in newly diagnosed glioblastoma has seen the final patient complete drug treatment; a number of patients remain in follow-up.
EVT801 phase I study protocol has been submitted to the French regulatory agency for review.
Kazia CEO, Dr James Garner, commented, "Kazia has seen an exceptionally busy first half, with excellent progress across our clinical programs. In particular, the GBM AGILE study is performing ahead of our forecasts in terms of recruitment. As we move into the second half of the year, we anticipate conclusion of the paxalisib phase II study, initial data readouts from a number of the paxalisib investigator-initiated studies in other forms of brain cancer, and commencement of the first-in-human phase I study of EVT801."

GBM AGILE

Almost twenty-five sites are currently open to the paxalisib arm in the United States. The list includes prestigious centres such as Memorial Sloan Kettering Cancer Center, Henry Ford Cancer Institute, Columbia University Irving Cancer Research Center, Emory University Winship Cancer Institute, and the University of Florida. The first site in Canada is expected to open in August 2021, followed by the first European sites in Q4 CY2021.

To date, GBM AGILE has screened over 650 patients. This progress is expected to accelerate as new sites in new territories come on stream.

GBM AGILE is an international platform study design to identify effective therapies for glioblastoma. It is an adaptive study, that evaluates multiple therapies in parallel, and recruits only the number of patients needed in each arm to reach a definitive answer. With its innovative design and efficient operational infrastructure, GBM AGILE is faster and more cost-effective than conventional company-sponsored approaches, and data from GBM AGILE may be used as the foundation of a new drug application to FDA and other regulatory agencies. GBM AGILE has been designed and implemented by the Global Coalition for Adaptive Research, a world-leading consortium focused on adaptive clinical trials.

To date, three experimental therapies have joined GBM AGILE: Bayer’s regorafenib, Kazia’s paxalisib, and Kintara Therapeutics’ VAL-083. It is expected that paxalisib will recruit up to 200 patients in the study, with the actual number determined by emergent data from the study.

Kazia has brought forward the manufacture of an additional batch of paxalisib investigational product, and this is expected to be released for use in the study during early 4Q CY2021.

Paxalisib Phase II Study

The final patient in the paxalisib phase II study has experienced disease progression after approximately 2.3 years on treatment. A number of patients remain in follow-up and the study is expected to conclude in 2H CY2021. Kazia anticipates no further interim analyses at this stage, and instead expects to release final data once the necessary analyses are complete.

Paxalisib Investigator-Initiated Studies

Kazia has been advised of generally good progress across the ongoing investigator-initiated studies of paxalisib in other forms of brain cancer. As previously indicated, the company expects that the respective investigators will release initial data from several of these studies during CY2021.

EVT801 Phase I Study

The study protocol for a planned phase I study of EVT801 has been submitted to ANSM, the French regulatory agency. Kazia expects to receive feedback from the agency late in 3Q CY2021.

The initial batch of EVT801 investigational product has been manufactured and is ready for use. Two leading academic research hospitals in France have been selected as the initial trial sites, with the potential to expand to additional sites as the study progresses.